Sarepta Therapeutics (NasdaqGS:SRPT) recently provided updates that may have influenced its stock performance. The announcement of a safety update for ELEVIDYS, concerning severe side effects and a ...

Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...

Writing in JAMA, four former government officials warn that the Trump administration’s involvement in delaying the approval ...

Edgewise Therapeutics (NASDAQ:EWTX) lost ~9% in the morning hours on Thursday after the company posted data from clinical ...

The agency is considering further regulatory action after a second Duchenne patient died from acute liver failure less than two months after receiving the gene therapy.

Investing.com -- Edgewise Therapeutics Inc (NASDAQ: EWTX) stock tumbled 7.2% after the company revealed that the U.S. Food and Drug Administration (FDA) deemed its CANYON trial data insufficient for ...

The FDA has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne ...

The MESA data demonstrated sustained disease stabilization, reinforcing prior ARCH and CANYON findings. Importantly, CANYON ...

The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

The FDA is investigating two deaths in non-ambulatory Duchenne muscular dystrophy patients who received a Sarepta gene ...

Duchenne muscular dystrophy is characterized by a mutation in the DMD gene that leads to a lack of dystrophin and muscle loss ...